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Novel therapeutic agents provide hope for patients with hard-to-treat blood disorders
Date:12/9/2012

apy or Hematopoietic Stem Cell Transplantation [Abstract 673]

Research suggests that a new targeted therapy, quizartinib, may be a safe and effective option to treat a subset of patients with treatment-resistant acute myeloid leukemia (AML).

AML is a fast-growing blood cancer in which patients produce an excessive amount of abnormal, immature white blood cells that are unable to adequately fight infection. Following AML diagnosis, leukemia cells from patients undergo genetic testing to identify the mutation driving the disease, which helps determine the appropriate treatment protocol. Of the many types of genetic mutations that can occur in AML, one of the most threatening is the FLT3-ITD (internal tandem duplication), which makes the leukemia even more aggressive and typically leads to failure of standard chemotherapy treatment response.

"The FLT3 mutation is essentially a power switch that leukemia cells use to spread more aggressively, which helps them to grow back immediately after chemotherapy," said Mark J. Levis, MD, PhD, lead author and Associate Professor of Oncology, Pharmacology, and Medicine at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Medicine in Baltimore. "The only way to treat this type of mutation is to find a way to turn the switch off a feat that has eluded researchers for far too long."

Patients with the FLT3-ITD mutation can achieve remission with standard chemotherapy but tend to relapse very quickly. An important treatment option is a stem cell transplant, but only if the patient is in some form of remission; otherwise, transplant failure rates are high. Given the barriers to treatment in this patient population, researchers have been examining the efficacy of the investigational oral agent quizartinib, which is designed to "turn off" the mutated FLT3 enzyme, thus forcing the immature cancer c
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Contact: Andrea Slesinski
aslesinski@hematology.org
614-352-5096
American Society of Hematology
Source:Eurekalert

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